000109054 001__ 109054 000109054 005__ 20211216151747.0 000109054 0247_ $$2doi$$a10.1111/apt.16181 000109054 0248_ $$2sideral$$a122745 000109054 037__ $$aART-2020-122745 000109054 041__ $$aeng 000109054 100__ $$aGomez, E. 000109054 245__ $$aEffectiveness and safety of obeticholic acid in a Southern European multicenter cohort of patients with primary biliary cholangitis and suboptimal response to ursodeoxycholic acid 000109054 260__ $$c2020 000109054 5060_ $$aAccess copy available to the general public$$fUnrestricted 000109054 5203_ $$aBackground Obeticholic acid (OCA) was recently approved as the only on-label alternative for patients with primary biliary cholangitis (PBC) with intolerance or suboptimal response to ursodeoxycholic acid (UDCA). However, few data are available outside clinical trials. Aim To assess the effectiveness and safety of OCA in a real-world cohort of patients with non-effective UDCA therapy. Methods Open-label, prospective, real-world, multicentre study, enrolling consecutive patients who did not meet Paris II criteria, from 18 institutions in Spain and Portugal. Effectiveness was assessed by the changes in GLOBE and UK-PBC scores from baseline. POISE and Paris II criteria were evaluated after 12 months of OCA . Liver fibrosis was evaluated by FIB-4 and AST to platelet ratio index (APRI). Results One hundred and twenty patients were eligible, median time since PBC diagnosis 9.3 (4.0-13.8) years, 21.7% had cirrhosis, and 26.7% received had previous or concomitant treatment with fibrates. Seventy-eight patients completed at least 1 year of OCA. The Globe-PBC score decreased to 0.17 (95% CI 0.05 to 0.28; P = 0.005) and the UK-PBC score decreased to 0.81 (95% CI -0.19 to 1.80; P = 0.11). There was a significant decrease in alkaline phosphatase of 81.3 U/L (95% CI 42.5 to 120; P < 0.001), ALT 22.1 U/L (95% CI 10.4 to 33.8; P < 0.001) and bilirubin 0.12 mg/dL (95% CI 0 to 0.24; P = 0.044). FIB-4 and APRI remained stable. According to the POISE criteria, 29.5% (23 out of 78) achieved response. The adverse events rate was 35%; 11.67% discontinued (8.3% due to pruritus). Conclusions This study supports data from phase III trials with significant improvement of PBC-Globe continuous prognostic marker score among OCA-treated patients with good tolerability. 000109054 540__ $$9info:eu-repo/semantics/openAccess$$aAll rights reserved$$uhttp://www.europeana.eu/rights/rr-f/ 000109054 590__ $$a8.171$$b2020 000109054 591__ $$aPHARMACOLOGY & PHARMACY$$b13 / 275 = 0.047$$c2020$$dQ1$$eT1 000109054 591__ $$aGASTROENTEROLOGY & HEPATOLOGY$$b15 / 92 = 0.163$$c2020$$dQ1$$eT1 000109054 592__ $$a3.307$$b2020 000109054 593__ $$aGastroenterology$$c2020$$dQ1 000109054 593__ $$aPharmacology (medical)$$c2020$$dQ1 000109054 593__ $$aHepatology$$c2020$$dQ1 000109054 655_4 $$ainfo:eu-repo/semantics/article$$vinfo:eu-repo/semantics/acceptedVersion 000109054 700__ $$aBuey, L.G. 000109054 700__ $$aMolina, E. 000109054 700__ $$aCasado, M. 000109054 700__ $$aConde, I. 000109054 700__ $$aBerenguer, M. 000109054 700__ $$aJorquera, F. 000109054 700__ $$0(orcid)0000-0003-2041-3147$$aSimon, M.A.$$uUniversidad de Zaragoza 000109054 700__ $$aOlveira, A. 000109054 700__ $$aHernandez-Guerra, M. 000109054 700__ $$aMesquita, M. 000109054 700__ $$aPresa, J. 000109054 700__ $$aCosta-Moreira, P. 000109054 700__ $$aMacedo, G. 000109054 700__ $$aArenas, J.I. 000109054 700__ $$aSousa, J.M. 000109054 700__ $$aAmpuero, J. 000109054 700__ $$aMorillas, R.M. 000109054 700__ $$aSantos, A. 000109054 700__ $$aDe Carvalho, A. 000109054 700__ $$aUriz, J. 000109054 700__ $$aCarrion, J.A. 000109054 700__ $$aGutierrez, M.L. 000109054 700__ $$aPerez-Fernandez, E. 000109054 700__ $$aFernandez-Rodriguez, C.M. 000109054 7102_ $$11007$$2610$$aUniversidad de Zaragoza$$bDpto. Medicina, Psiqu. y Derm.$$cArea Medicina 000109054 773__ $$g53, 4 (2020), 519-530$$pAliment. pharmacol. ther.$$tALIMENTARY PHARMACOLOGY & THERAPEUTICS$$x0269-2813 000109054 8564_ $$s638999$$uhttps://zaguan.unizar.es/record/109054/files/texto_completo.pdf$$yPostprint 000109054 8564_ $$s2379913$$uhttps://zaguan.unizar.es/record/109054/files/texto_completo.jpg?subformat=icon$$xicon$$yPostprint 000109054 909CO $$ooai:zaguan.unizar.es:109054$$particulos$$pdriver 000109054 951__ $$a2021-12-16-12:54:46 000109054 980__ $$aARTICLE